Friday February 11, 7:32 am Eastern Time
Company Press Release
FDA Approval of Actimmune Provides Hope for
Children With Fatal Disease
PALO ALTO, Calif.--(BW HealthWire)--Feb. 11, 2000--InterMune Pharmaceuticals
today announced that the U.S. Food and Drug Administration has approved Actimmune®
(Interferon gamma-1b) Injection for delaying the time to disease progression in patients with
severe, malignant osteopetrosis. Osteopetrosis is a life-threatening, congenital disorder in which an overgrowth of bony
structures leads to blindness, deafness and increased susceptibility to infections. In the most serious form of the disease, most
patients become blind or anemic by six months of age and die within the first ten years of life, frequently in the first two years.
Actimmune was previously approved in 1990 for reducing the frequency and severity of serious infections associated with
Chronic Granulomatous Disease.
''Actimmune provides us with a new treatment option for osteopetrosis in that it helps to reduce the number of infections,
improve bone marrow function and prolong the lives of children with this disease,'' said L. Lyndon Key, Jr., M.D., Professor of
Pediatric Endocrinology at the Medical University of South Carolina.
''The approval of Actimmune is a significant milestone for InterMune and for patients who suffer from osteopetrosis as it makes
available a much needed medication to patients in desperate need of treatment alternatives,'' said W. Scott Harkonen, M.D.,
President and Chief Executive Officer of InterMune Pharmaceuticals. ''Additionally, we believe that Actimmune has a
tremendous potential in the treatment of a variety of other diseases. In fact, published clinical trial results have suggested that the
drug may be useful in treating serious lung diseases and systemic infections.''
Actimmune In The Treatment of Osteopetrosis
The FDA's decision to approve Actimmune (Interferon gamma-1b) Injection was based on a BLA application filed in August
1999. The Phase III clinical trial evaluated the efficacy of Actimmune by determining the length of time to disease progression.
Disease progression was defined as death, significant reduction in hemoglobin or platelet counts, serious bacterial infection
requiring antibiotics, a 50 decibel decrease in hearing or progressive optic atrophy. In the trial, 15 patients were randomized to
receive either Actimmune or a control vitamin D medication called calcitriol. Study results showed that the median time to
disease progression was significantly delayed in patients treated with Actimmune (at least 165 days) compared to patients
treated with the control (65 days). All side effects seen in patients who participated in both the Phase II and Phase III clinical
trials (34 patients) were mild in severity, the most common being flu-like symptoms such as fever (16 patients), headache (3
patients), and fatigue (1 patient) which could be alleviated by taking acetaminophen one hour prior to injection.
Prior to the filing, the FDA granted the drug fast track review and orphan drug status. The FDA's fast track program facilitates
the development and expedites the review of drugs intended for the treatment of serious or life-threatening diseases and that
demonstrate the potential to address unmet medical needs for such conditions. The FDA may grant orphan drug designation to
drugs intended to treat a rare disease or condition. Once approved, the FDA may not approve any other applications to
market the same drug for the same indication, except in very limited circumstances, for seven years.
Actimmune Injection
Interferon gamma-1b, the active ingredient in Actimmune, is a human protein which is a potent stimulator of the immune system.
Interferon gamma is biologically distinct from Interferon alpha and Interferon beta, two related proteins that are currently
marketed for the treatment of diseases such as hepatitis B infection and multiple sclerosis.
InterMune Pharmaceuticals, Inc. develops and commercializes innovative products for the treatment of serious pulmonary and
infectious diseases and congenital disorders. The Company currently markets Actimmune® (Interferon gamma-1b) Injection in
the U.S. for the treatment of Chronic Granulomatous Disease (CGD).
Complete prescribing information is available upon request at our website: www.actimmune.com, or by telephone at
1-888-696-8036.
Contact:
InterMune Pharmaceuticals, Palo Alto
W. Scott Harkonen, M.D., President and CEO, 650/843-2855
or
Burns McClellan, New York
Jonathan M. Nugent (investors),
Lisa Fern (media),
212/213-0006 |
